Positive Phase III data from PLEO-CMT trial for PXT3003 (baclofen, naltrexone and sorbitol oral solution) in Charcot-Marie-Tooth disease Type 1a

In RCT in 323 patients with this condition, characterised by chronic peripheral neuropathies, high-dose PXT3003 associated with statistically significant improvement in Overall Neuropathy Limitations Scale v placebo (mean difference -0.37 points; 97.5% CI -0.68 to -0.06; p=0.008)

SPS commentary:

The European Medicines Agency and US FDA have requested a second Phase III trial be conducted as a result of premature discontinuation of the high-dose group due to an unexpected crystal formation in the high-dose formulation. Results from this trial are expected to be announced in 2023.

Biospace Inc.